CRISPR Therapy in LCA Patients Shows Positive Results

Article: A Gene-Editing Experiment Let These Patients With Vision Loss See Color Again
Source: NPR
Published: September 29, 2021

Scientists report encouraging results for the first few cases of CRISPR gene-editing for Leber congenital amaurosis (LCA), presenting their findings at the International Symposium on Retinal Degeneration. The gene-editing was notable in this case for being the first trial to inject CRISPR in vivo in human subjects, as compared to explanting cells, editing them in vitro, and then infusing them back into tissues of the body. Thus far, seven patients have volunteered to have the experimental therapy. NPR interviewed two of those patients, Carlene Knight and Michael Kalberer. Knight reports, "I was bumping into the cubicles and really scaring people that were sitting at them." With vision improved enough to make out doorways, navigate hallways, spot objects and even see colors, she says she no longer scares people and has fewer bruises from bumping into things. Knight also says that colors are more vivid, which she has appreciated since she was a kid. "I've always loved colors. Since I was a kid it's one of those things I could enjoy with just a small amount of vision. But now I realize how much brighter they were as a kid because I can see them a lot more brilliantly now," she says. Similarly, Kalberer reports being thrilled at his improved vision, which he noticed starting one month after the treatment. For example, he is now able to recognize shapes and light much better, and has regained more peripheral vision. It was an especially joyous moment for him to watch the DJ's strobe lights change color at his cousin's wedding and seeing the sunset again for the first time. One of the clinical researchers at HMS says, "We're thrilled about this. This is the first time we're having evidence that gene editing is functioning inside somebody and it's improving — in this case — their visual function." They next plan on trials at higher doses, and in age brackets that have the best chance of benefiting. Although the treatment is far from a cure, vision never returned to normal, and visual improvement is not seen in all seven patients at this point, for some patients in the trial, the changes experienced are enough to have a meaningful impact on their daily lives.

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